Also known as: Translarna
Ataluren is a small-molecule medicine developed for nonsense mutation Duchenne muscular dystrophy and marketed as Translarna. It was conditionally authorized in the EU in 2014 for ambulatory patients with dystrophin nonsense mutations, but the European Commission decided on March 28, 2025 not to renew the authorization. As of that decision, the EU authorization is expired.
PTC Therapeutics has withdrawn its New Drug Application resubmission for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy after the FDA indicated the submission lacked substantial evidence of effectiveness.
PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.
PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds, ending a pursuit spanning more than a decade.
PTC Therapeutics has withdrawn its New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy following FDA feedback that the data are unlikely to meet the threshold for substantial evidence of effectiveness.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT04336826 |
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) |
COMPLETED | PHASE2 |
| NCT04117880 |
A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia |
WITHDRAWN | PHASE2 |
| NCT03796637 |
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren |
COMPLETED | PHASE2 |
| NCT03648827 |
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) |
COMPLETED | PHASE2 |
| NCT03256968 |
PTC Study to Evaluate Ataluren in Combination With Ivacaftor |
COMPLETED | PHASE4 |
| NCT03179631 |
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy |
COMPLETED | PHASE3 |
| NCT02819557 |
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy |
COMPLETED | PHASE2 |
| NCT02758626 |
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome |
COMPLETED | PHASE2 |
| NCT02647359 |
Study of Ataluren in Participants With Nonsense Mutation Aniridia |
COMPLETED | PHASE2 |
| NCT02456103 |
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis |
TERMINATED | PHASE3 |
